AveXis Launches Spinal Muscular Atrophy Clinical Trials Website

Ana de Barros, PhD avatar

by Ana de Barros, PhD |

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AveXisDallas, Texas-based AveXis, Inc., a biotechnology company working to improve the quality of life of patients suffering from severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA) through the use of gene therapy, is launching a new website specifically dedicated to SMA, called SMAStudy.org. Focused on direct communication with families, the website is part of the company’s efforts to raise awareness about the disease and its current chariSMA clinical trial.

While the company already has a corporate website, the new site is being launched in order to provide information on ongoing and upcoming clinical trials of their investigational drugs, designed for the parents of children suffering from SMA who are seeking options to fight the disease.

“It is very important for AveXis to remain rooted and communicate effectively to the community which is so supportive of our work in gene therapy,” said CEO of AveXis, John A. Carbona. “The intention of this new website is to empower people around the world to learn about SMA, to inform them of their clinical trial options, and to provide links to educational support organizations, which are always useful when looking for answers.”

The company’s current focus is on recruiting participants for chariSMA, which is a Gene Transfer Clinical Trial for SMA Type 1 (NCT02122952) that is currently open for patient enrollment. AveXis is providing detailed information about the investigational drug on the website, as the company hopes to associate patients, parents, and families with organizations, foundations, and support and advocacy groups worldwide to test their experimental SMA therapy.

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ChariSMA, an intravenous therapy that delivers a genetically-modified virus comprised of the Survival Motor Neuron replacement gene (SMN) through AveXis’s ReGenX Biosciences technology platform, is currently being tested in collaboration with Nationwide Children’s Hospital in Columbus, Ohio, which launched patient enrollment last April. The trial will assess the safety and efficacy of the therapy, which is the first human gene treatment in the world for pediatric SMA. The company expects to improve survival rate and motor function of dosed SMA patients, and has recently finished dosing patients in a previous low dose cohort phase 1 non-randomized clinical trial.

In addition, the company has received orphan drug designation from the U.S. Food and Drug Administration, which allows for accelerated development of novel therapies for the treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. The company is also providing links to other SMA-related clinical trials at SMAStudy.org.